- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05626855
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (ONYX)
April 10, 2024 updated by: Scholar Rock, Inc.
An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
260
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Desiree Warne
- Phone Number: 857-259-3860
- Email: dwarne@scholarrock.com
Study Contact Backup
- Name: Paul Miller
- Phone Number: 857-259-3860
- Email: pmiller@scholarrock.com
Study Locations
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Gent, Belgium, 9000
- UZ Gent
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Contact:
- Elke de Vos
- Phone Number: (+32) 9 332 19 54
- Email: elke.devos@uzgent.be
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Leuven, Belgium, 3000
- UZ Leuven
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Contact:
- Scholar Rock Clinical Trials
- Phone Number: 833-724-7625
- Email: clinicaltrials@scholarrock.com
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Liege, Belgium, 4000
- CHR Citadelle
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Lille, France, 59037
- CHRU de Lille - Hpital Jeanne de Flandre
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Paris, France, 75012
- Hopital Trousseau - I-Motion
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Toulouse, France, 31059
- CHU Toulouse Hopital des Enfants
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Essen, Germany, 45147
- Universitätsklinikum Essen
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Freiburg, Germany, 79106
- Universitätsklinikum Freiburg
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Milano, Italy, 20162
- NeuroMuscular Omnicentre
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Milano, Italy, 20133
- Carlo Besta Neurological Research Institute
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Roma, Italy, 106
- Fondazione Policlinico Universitario A. Gemelli
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Utrecht, Netherlands, 3508
- UMC Utrecht
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Gdańsk, Poland, 80-211
- Uniwersyteckie Centrum Kliniczne
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Poznań, Poland, 60-355
- Uniwersytecki Szpital Kliniczny w Poznaniu, Oddział Kliniczny Neurologii Dzieci i Młodzieży
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Warsaw, Poland, 04-730
- Instytut Pomnik - Centrum Zdrowia Dziecka
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Barcelona, Spain, 08950
- Hospital Sant Joan de Deau
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Valencia, Spain, 46026
- Hospital Universitari I Politecnic La Fe
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Leeds, United Kingdom, LS1 3EX
- Leeds Children's Hospital Clinical Research
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Arizona
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Phoenix, Arizona, United States, 85016
- Phoenix Childrens Hospital
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California
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La Jolla, California, United States, 92037
- UCSD Altman Clinical and Translational Research
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Palo Alto, California, United States, 94304
- Stanford Neuroscience Health Center
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Contact:
- Allison Stone
- Phone Number: 720-777-0058
- Email: neuromuecularresearch@childrenscolorado.org
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Florida
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Orlando, Florida, United States, 32827
- Nemours Biomedical Research
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Illinois
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Chicago, Illinois, United States, 60611
- Lurie Children's Hospital of Chicago
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa
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Kansas
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Fairway, Kansas, United States, 66205
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, United States, 21287
- Johns HopkinsHospital
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Michigan
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Grand Rapids, Michigan, United States, 49503
- Helen DeVos Children's Hospital
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Contact:
- Rosa Sifontes-Escamilla
- Phone Number: 616-486-6342
- Email: rosa.sifontes-escamilla@helendevoschildrens.org
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Minnesota
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Saint Paul, Minnesota, United States, 55101
- Gillette Children's Specialty Healthcare
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Contact:
- Katherine Harrison
- Phone Number: 651-578-5223
- Email: KatherineAHarrison@gillettechildrens.com
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University Medical Campus
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New York
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New York, New York, United States, 10032
- Columbia University Medical Center
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North Carolina
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest University School of Medicine
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Ohio
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Columbus, Ohio, United States, 43215
- Nationwide Children's Hospital
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health & Science University
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Tennessee
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Memphis, Tennessee, United States, 38105
- St. Jude Children's Research Hospital
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Contact:
- Jean Laboe
- Phone Number: 901-595-1693
- Email: jean.laboe@stjude.org
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Texas
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Dallas, Texas, United States, 75207
- University of Texas Southwestern - Pediatric Neurology
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Virginia
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Newport News, Virginia, United States, 23606
- Children's Specialty Group PLLC (Children's Hospital of The King's Daughters)
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Washington
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Seattle, Washington, United States, 98115
- Seattle Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
7 months and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Patients have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial. (For TOPAZ, completed is defined as completion of Visit EC14 in Extension Period C or participating in TOPAZ at the time the trial is ended. For SAPPHIRE, completed is defined as completion of Visit 14 or participating in SAPPHIRE at the time the trial is ended)
- Estimated life expectancy >2 years from Baseline (Day 1)
- Able to receive study drug infusions and provide blood samples through the use of a peripheral IV or a long-term IV access device that the patient has placed for reasons independent from the trial
- Able to adhere to the requirements of the protocol, including travel to the trial site and completing all trial procedures and trial visits
- Females of childbearing potential must have a negative pregnancy test at Baseline and agree to use at least 1 acceptable method of contraception throughout the trial and for 20 weeks after the last dose of apitegromab
Exclusion Criteria:
- Patient permanently discontinued study treatment during the feeder trial (i.e., TOPAZ or SAPPHIRE)
- Nutritional status that was not stable over the past 6 months and is not anticipated to be stable throughout the trial or medical necessity for a gastric/nasogastric feeding tube, where the majority of feeds are given by this route, as assessed by the Investigator
- Patient is currently enrolled in any investigational drug trial other than TOPAZ or SAPPHIRE
- Prior history of severe hypersensitivity reaction or intolerance to SMN-targeted therapies
- Prior history of severe hypersensitivity reaction or intolerance to apitegromab
- Use of chronic daytime noninvasive ventilatory support for >16 hours daily in the 2 weeks before dosing, or anticipated to regularly receive such daytime ventilator support chronically throughout the trial
- Any acute or comorbid condition interfering with the well-being of the patient at the patient's last visit in TOPAZ or SAPPHIRE, (including active systemic infection, the need for acute treatment, or inpatient observation due to any reason). After resolution of the condition, the patient can be enrolled in the trial if they meet all the other eligibility criteria.
- Pregnant or breastfeeding
- Any other condition or clinically significant laboratory result or ECG value that, in the opinion of the Investigator, may compromise safety or compliance, would preclude the patient from successful completion of the trial, or interfere with the interpretation of the results
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Treatment Period
Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period
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Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
Time Frame: Up to 6 years
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Incidence of TEAEs and SAEs by severity
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Up to 6 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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Hammersmith Functional Motor Scale Expanded (HFMSE) total score at prespecified time points (excludes ambulatory patients)
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Up to 6 years
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Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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Revised Upper Limb Module (RULM) total score at prespecified time points (excludes ambulatory patients)
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Up to 6 years
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Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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Number of World Health Organization (WHO) motor development milestones attained at prespecified time points (excludes ambulatory patients)
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Up to 6 years
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Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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Revised Hammersmith Scale (RHS) total score
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Up to 6 years
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Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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Results for 6-Minute Walk Test
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Up to 6 years
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Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
Time Frame: Up to 6 years
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30-Second Sit-to-Stand
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Up to 6 years
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Further evaluate the immunogenicity of apitegromab
Time Frame: Up to 6 years
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Presence or absence of antidrug antibody (ADA) against apitegromab in serum from blood samples
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Up to 6 years
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Further characterize the PK of apitegromab
Time Frame: Up to 6 years
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Apitegromab concentrations in serum from blood samples at prespecified time points
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Up to 6 years
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Further evaluate the pharmacodynamic (PD) effects of apitegromab
Time Frame: Up to 6 years
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Circulating latent myostatin concentrations in blood samples at prespecified time points
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Up to 6 years
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To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior
Time Frame: Up to 6 years
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Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) at prespecified time points
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Up to 6 years
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To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior
Time Frame: Up to 6 years
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Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue Questionnaire at prespecified time points
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Up to 6 years
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To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior
Time Frame: Up to 6 years
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Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) at prespecified time points
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Up to 6 years
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To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior
Time Frame: Up to 6 years
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Columbia-Suicide Severity Rating Scale (C-SSRS) at prespecified time points
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Up to 6 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 17, 2023
Primary Completion (Estimated)
November 1, 2026
Study Completion (Estimated)
January 1, 2027
Study Registration Dates
First Submitted
November 7, 2022
First Submitted That Met QC Criteria
November 15, 2022
First Posted (Actual)
November 25, 2022
Study Record Updates
Last Update Posted (Actual)
April 12, 2024
Last Update Submitted That Met QC Criteria
April 10, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Pathological Conditions, Anatomical
- Spinal Cord Diseases
- Heredodegenerative Disorders, Nervous System
- Motor Neuron Disease
- Muscular Atrophy
- Atrophy
- Muscular Atrophy, Spinal
- Neuromuscular Manifestations
- Neuromuscular Diseases
- Spinal Muscular Atrophies of Childhood
Other Study ID Numbers
- SRK-015-004
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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